Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia for ≥6 Months
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| ClinicalTrials.gov Identifier: NCT04516967 |
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Recruitment Status :
Recruiting
First Posted : August 18, 2020
Last Update Posted : March 24, 2023
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Sponsor:
Sobi, Inc.
Information provided by (Responsible Party):
Sobi, Inc.
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Brief Summary:
A Phase 3b Study to Evaluate the Efficacy and Safety of Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects with Immune Thrombocytopenia for ≥6 Months
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Immune Thrombocytopenia | Drug: Avatrombopag Drug: Placebo | Phase 3 |
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 72 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | Double (Participant, Investigator) |
| Primary Purpose: | Treatment |
| Official Title: | Randomized, Double-blind, Placebo-controlled Study With Open-label Extension Phase to Evaluate the Efficacy and Safety of Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia for ≥6 Months |
| Actual Study Start Date : | March 2, 2021 |
| Estimated Primary Completion Date : | June 2, 2023 |
| Estimated Study Completion Date : | November 28, 2025 |
Resource links provided by the National Library of Medicine
| Arm | Intervention/treatment |
|---|---|
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Active Comparator: Experimental: Avatrombopag
Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks
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Drug: Avatrombopag
Oral avatrombopag Tablet
Other Name: Doptelet |
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Placebo Comparator: Placebo Comparator:Placebo
Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks
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Drug: Placebo
Placebo comparator tablet |
Primary Outcome Measures :
- Durable platelet response [ Time Frame: Last 8 weeks of 12 week treatment regimen ]The proportion of subjects achieving at least 6 out of 8 weekly platelet counts ≥50×10^9/L during the last 8 weeks of the 12 week Treatment Period in the Core Phase, in the absence of rescue medication
Secondary Outcome Measures :
- Platelet Counts [ Time Frame: 12 weeks of treatment ]The percentage of weeks subjects have a platelet count ≥50×10^9/L during 12 weeks of treatment in the Core Phase, in the absence of rescue therapy.
- Platelet response [ Time Frame: Day 8 ]The proportion of subjects with a platelet count ≥50×10^9/L at day 8, in the absence of rescue therapy.
- Platelet percentage [ Time Frame: 12 weeks ]The percentage of weeks subjects have a platelet count between ≥50×10^9/L and ≤150×10^9/L, during 12 weeks of treatment in the Core Phase, in the absence of rescue therapy.
- Rescue medications [ Time Frame: 12 weeks ]The proportion of subjects who require rescue medications during 12 weeks of treatment in the Core Phase of the study.
- Bleeding Symptoms [ Time Frame: Throughout study ]Incidence and severity of bleeding symptoms associated with Immune thrombocytopenia (ITP) measured using the World Health Organization (WHO) Bleeding Scale.
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| Ages Eligible for Study: | 1 Year to 17 Years (Child) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Male or female participants ≥1 and <18 years of age at Screening and Baseline with a diagnosis of primary ITP for ≥6 months duration and has had an insufficient response to a previous treatment, in the opinion of the Investigator.
- Participant has an average of 2 platelet counts <30×10^9/L with no single count >35×10^9/L in the screening period
Exclusion Criteria:
- Participants must not have a known history of secondary ITP, any history of arterial or venous thrombosis, including partial or complete thrombosis, known inherited thrombocytopenia, a history of myelodysplastic syndrome (MDS) or known history of congenital heart abnormalities or arrhythmias.
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04516967
Contacts
| Contact: Sobi Inc | 919-765-6671 | NAClincal@Sobi.com |
Locations
Show 62 study locations
Sponsors and Collaborators
Sobi, Inc.
| Responsible Party: | Sobi, Inc. |
| ClinicalTrials.gov Identifier: | NCT04516967 |
| Other Study ID Numbers: |
AVA-PED-301 |
| First Posted: | August 18, 2020 Key Record Dates |
| Last Update Posted: | March 24, 2023 |
| Last Verified: | March 2023 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
Keywords provided by Sobi, Inc.:
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ITP |
Additional relevant MeSH terms:
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Thrombocytopenia Purpura, Thrombocytopenic, Idiopathic Blood Platelet Disorders Hematologic Diseases Purpura, Thrombocytopenic Purpura Blood Coagulation Disorders |
Thrombotic Microangiopathies Hemorrhagic Disorders Autoimmune Diseases Immune System Diseases Hemorrhage Pathologic Processes Skin Manifestations |